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Dec. 11, 2012 -- An inexpensive generic drug may ease autism in children, a small new study shows.

The drug, bumetanide, is a diuretic, or a drug that rids the body of extra water through urine. It’s been FDA-approved since 1983 to reduce fluid buildup in patients with heart failure, kidney disease, or liver disease.

In autism, bumetanide may help to correct a chemical imbalance in the brain, says Jing-Qiong Kang, MD, PhD, an assistant professor of neurology at Vanderbilt University in Nashville, Tenn. Kang studies the biology of autism, but she was not involved in the current research.

Unlike other medications, which are used to quell behavioral problems that are sometimes associated with autism, like hyperactivity, insomnia, and aggression, bumetanide seems to ease some of the core features of the condition, including problems connecting and interacting with other people.

“They were making more eye contact, more spontaneous speech, and more two-way conversation,” says Daniel Coury, MD, a pediatric psychiatrist in Columbus, Ohio, who is medical director for the Autism Speaks Autism Treatment Network.

“At the end of the day parents said, ‘I like this. My child is doing better,’” says Coury, who was not involved in the research.

The Research

For the study, French researchers had 60 children with autism take either bumetanide or a look-alike placebo pill for 90 days.

Children in the study ranged from 3 to 11 years old. Their symptoms were scored by clinicians who did not know whether they were taking the drug or the placebo. Parents and teachers were also asked about changes in the children’s behavior over time.

By the end of the study, children in the treatment group shifted from average autism scores in the severe range to the mild or moderate range.

“A third of the placebo group improved, but three-fourths of the treatment group improved. So that suggests that it seems to be a real finding there,” Coury says.

The study is published in the journal Translational Psychiatry.

More Research Needed

Despite the hopeful news, Coury and other experts urged caution.

“Is it going to have a sustained improvement, a continued improvement? There’s a lot that we don’t know,” he says.

About 30% of the children in the study developed low potassium levels, which were reversed with supplements. Two children stopped the study because of bedwetting. One of them was taking the placebo.

At the end of the study, researchers say most parents asked to continue on the bumetanide treatment. Researchers report that 87 children are now taking the medication. Some have now been on it for as long as two years.

Researcher Yehezkel Ben-Ari, PhD, director of research at the National Institute of Health and Medical Research in Marseille, France, says the medication seems to help children with milder forms of autism spectrum disorders, like Asperger’s syndrome, the most.

Since the completion of the study, Ben-Ari has become the CEO and a major shareholder of a company that’s seeking to develop and market bumetanide as a treatment for autism.

“We don’t have any medicines that remedy autism, even if we don’t cure it. So bumetanide is an interesting and promising medication,” says Andrew Adesman, MD, chief of developmental & behavioral pediatrics at the Cohen Children's Medical Center in New Hyde Park, N.Y. Adesman treats patients with autism, but he was not involved in the research.

Because the medication is already on the market and seems to be relatively safe, Adesman said he expects doctors and families may be tempted to try it right away.

“As difficult as it may be, families and professionals are probably best off waiting for a little bit more research,” he says.